title: Superselective intra-arterial melphalan therapy for newly diagnosed and refractory retinoblastoma: results from a single institution.
abstract: BACKGROUND
  Intra-arterial administration of melphalan chemotherapy has shown promise in the treatment of retinoblastoma. This report describes our results using superselective intra-arterial melphalan in patients with newly diagnosed retinoblastoma and those who were treated for progression after systemic chemotherapy.
  
  
  METHODS
  This is a retrospective review of all retinoblastoma patients treated with intra-arterial melphalan at the University of California, San Francisco from March 2010 to August 2012. Twenty eyes (16 patients) underwent 40 intra-arterial melphalan infusions, and dose was determined by age. Patients were treated at monthly intervals and received a range of 1-5 treatments. Response to therapy, toxicity, and procedural radiation exposure was assessed.
  
  
  RESULTS
  All patients are alive without metastatic disease at a median follow-up of 14.5 (1-29) months. Treatment with enucleation or external beam radiation was avoided in 11/20 eyes (55%) overall [6/12 (50%) in newly diagnosed eyes and 5/8 (63%) in refractory/relapsed eyes]. Response rates (per the International Classification of Retinoblastoma) were as follows: 6/7 (86%) in groups A-C and 5/13 (38%) in groups D and E. Nonhematologic and hematologic toxicities were minimal and comparable with those in previous reports. The mean procedural radiation dose was 20.2 ± 11.9 mGy per eye per procedure.
  
  
  CONCLUSIONS
  Superselective intra-arterial melphalan therapy is effective for less advanced eyes but further modifications to therapy are required to improve results in eyes with advanced retinoblastoma.
authors.affiliation: Department of Pediatrics, University of California, San Francisco School of Medicine, San Francisco, CA, USA.
authors.forename: Sheila
authors.initials: S
authors.lastname: Thampi
authors.forename: Steven W
authors.initials: SW
authors.lastname: Hetts
authors.forename: Daniel L
authors.initials: DL
authors.lastname: Cooke
authors.forename: Paul J
authors.initials: PJ
authors.lastname: Stewart
authors.forename: Elizabeth
authors.initials: E
authors.lastname: Robbins
authors.forename: Anuradha
authors.initials: A
authors.lastname: Banerjee
authors.forename: Steven G
authors.initials: SG
authors.lastname: Dubois
authors.forename: Devron
authors.initials: D
authors.lastname: Char
authors.forename: Van
authors.initials: V
authors.lastname: Halbach
authors.forename: Katherine
authors.initials: K
authors.lastname: Matthay
keywords: intra-arterial; melphalan; retinoblastoma